This is a first-in-human (FIH) study of RG6496 that will assess the safety and tolerability of single-ascending doses of RG6496 administered to huntington's disease gene expansion carriers (HDGECs). The study consists of two parts: Part 1 \[single-ascending dose\] followed by Part 2 \[open-label extension (OLE)\].
Confirmation of HDGEC status with cytosine-adenine-guanine (CAG) expansion > 39.
Confirmation of SNP carrier status of the target SNP
Independence Scale (IS) score of ≥70, total functional capacity (TFC) ≥10, total motor score (TMS) >6.
Ability to read the words "red," "blue," and "green" and be fluent in the language of the informed consent form (ICF) and the tests used at the study site.
Concurrent or planned participation in any interventional clinical study, including current use of an ASO or any HTT-lowering therapy or treatment with investigational therapy within 90 days or 5 drug-elimination half-lives, whichever is longer prior to screening
Pregnant or breastfeeding, or with the intention of becoming pregnant during the study or within the timeframe in which contraception is required
Malignancy within 5 years prior to screening
Planned brain surgery during the study
Hoffmann-La Roche
Lead sponsor